Salvage Treatment of Adrenocortical Carcinoma with Trofosfamide.

摘要: Adrenocortical carcinoma (ACC) has a dismal prognosis in advanced stages. Despite treatment with the adrenal toxicant mitotane and/or aggressive chemotherapy, tumor control is often short-lived. Here, we examine trofosfamide as salvage of ACC an observational cohort study within German registry. Response defined progression-free survival (PFS) at first evaluation was assessed by RECIST 1.1 or clinically, and PFS overall (OS) were estimated Kaplan-Meier method. Twenty-seven patients (11 males; median age 46.9 years) progressing after three (median, range 0-5) other systemic treatments evaluated for safety. Trofosfamide (150 mg/day) administered monotherapy (n = 13) combination (n = 14). Overall tolerability good only mild adverse events. Six did not meet criteria response assessment. Of 21 patients, 8 had clinically progressive disease (3 deaths from ACC); among 13 evaluable 1.1, best to stable (SD, n = 3) (n = 10). Hence, predefined met 3/21 (14 %). Median 84 days (95 % confidence interval 74-95) OS 198 days CI 89-307). One prolonged stabilization (best -26 %) observed 479 days. In conclusion, well tolerated but rather rare. Accordingly, it may be used selected cases amenable options such clinical trials.